Create a Pediatric Immunotherapy Discovery and Development Network (PI-DDN)

Immunotherapy makes use of the immune system to recognize and destroy infected, damaged, and transformed cells. This type of cancer treatment enhances the activity of immune cells to attack tumors. While immunotherapeutic approaches have been used successfully to treat a limited number of childhood cancers, particularly leukemia and neuroblastoma, there are currently no effective immunotherapy options for most patients with high-risk or difficult-to-treat childhood cancers.

There are a number of barriers to the development of immunotherapies for childhood cancers. Unlike many adult tumors, many childhood cancers express few or no markers that can be recognized by the immune system. Similarly, many immunotherapies target specific markers that are expressed in adult cancers but not childhood cancers. Also, tumors that develop in children can develop microenvironments that suppress the immune system and reduce the effectiveness of immunotherapies.

This recommendation is aimed at creating a pediatric immunotherapy science network that will focus on a collaborative effort to discover targets for immunotherapies and develop and test new treatment approaches, including cancer vaccines, cellular therapy, and combinations of immunotherapy and other forms of therapy.

Ultimately, the hope is to develop new immunotherapies and improve the effectiveness of current immunotherapy treatments to reduce the burden of cancer in children.

The Pediatric Immunotherapy Discovery and Development Network (PI-DDN)

This collaborative network is identifying and advancing preclinical immunotherapy research for children and adolescents with cancer. The network is working to discover and characterize new targets for immunotherapies, design experimental models to test the effectiveness of pediatric immunotherapies, develop new pediatric immunotherapy treatments, and improve the understanding of tumor immunity in children and adolescents with cancer. PI-DDN is also working to overcome major barriers in developing effective immunotherapies for children and adolescents, such as lower levels of proteins in tumor cells that can be recognized by immune cells and the immunosuppressive environments of tumors in some pediatric cancers.

Investigators in the PI-DDN are working together on multicomponent research studies centered around a pediatric cancer research area and individual pediatric immunotherapy projects. The goal of the network is to advance pediatric immunotherapies to treat children and adolescents with high-risk cancers.

Highlights of PI-DDN Research Progress

A PI-DDN research team has made advances in understanding and overcoming immune cell exhaustion to improve the cancer-fighting ability of CAR T-cells.

PI-DDN researchers are also identifying molecules on the surface of cancer cells that can be targets of new immune-based therapies. For example, there have been promising results using CAR T cells that are engineered to target the GPC2 protein in pediatric brain tumors.

Another team of PI-DDN researchers identified immune signatures of acute myeloid leukemia (AML) that are predictive of chemotherapy and immunotherapy response and resistance. Understanding these AML subtypes could inform the delivery of personalized therapies for children with cancer.

The PI-DDN program has been instrumental in building relationships and collaborations within the network, with other Cancer Moonshot initiatives, and with the broader research community. The steering committee includes a patient advocate and other associate members in its meetings. Through the creation of this network, the field of immunotherapy and immunoprevention research for childhood cancers has expanded and accelerated. 

Pediatric Immunotherapy Projects Awarded Cancer Moonshot Funding